Project Grant W81XWH1810165

This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, supports preclinical development of a gene therapy approach using adeno-associated virus (AAV) containing U7snRNA to skip exon 44 in the Duchenne muscular dystrophy (DMD) gene. The goal is to restore the reading frame and express a truncated but functional dystrophin protein, which could...

The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $774,361 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to Loyola University of Chicago for preclinical development of novel site-blocking antisense oligonucleotides (SBASOs) to fine-tune expression of the MECP2 gene for the treatment of Rett syndrome. The research project aims to address the challenge of narrow dosage requirements for MECP2 gene...

This federal Project Grant award from the National Institute of Biomedical Imaging and Bioengineering (NIBIB), under the Discovery and Applied Research for Technological Innovations to Improve Human Health program (CFDA 93.286), is supporting research to develop an efficient synthetic RNA switch that can regulate the timing and dosing of gene therapies delivered via adeno-associated viral (AAV) vectors. The $1,158,262 award to the University of Massachusetts Medical School will fund the...

The U.S. Department of Health and Human Services, National Institute of Neurological Disorders and Stroke (NINDS) awarded a $429,000 Project Grant to Thomas Jefferson University, doing business as Sidney Kimmel Medical College, under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program. The funding supports research to develop a mechanistic understanding of Spinal and Bulbar Muscular Atrophy (SBMA) pathogenesis and explore therapeutic approaches....

This $438,365 Project Grant awarded by the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) supports research to investigate the role of the CD38 protein and its potential as a therapeutic target for Spinal and Bulbar Muscular Atrophy (SBMA). The research aims to use stem cell technology and CRISPR-corrected isogenic controls to examine metabolic alterations in SBMA patient-derived...

<p>This $660,000 Project Grant awarded by the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420) is funding Nucyrna Therapeutics Inc. to develop a SOD1-targeting oligonucleotide drug candidate with improved efficacy and safety compared to the existing drug tofersen. The award period runs from July 1, 2025 to June 30, 2027. The goal is to advance a novel therapeutic option for treating neurodegenerative conditions associated with the SOD1 gene.</p>

Acrigen Biosciences, Inc. was awarded a $1,000,000 cooperative agreement from the National Science Foundation under the Engineering program (CFDA 47.041) to develop a curative gene therapy for spinal muscular atrophy. Over the two year period from March 2022 to February 2024, Acrigen will engineer an anti-CRISPR protein to increase the precision and safety of CRISPR-Cas gene editing. They will pair this with a novel CRISPR nuclease and validate the system for editing and disease correction in...

This $499,300 Project Grant award from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) supports research by Myogene Bio LLC, a woman-owned small business in San Diego, California, to advance manufacturing and release testing of a gene editing therapy for Duchenne muscular dystrophy (DMD). The key products and services to be delivered include: 1) Optimizing the upstream...

Acurastem Inc. received a $839,898.40 Project Grant award from the Defense Health Agency under the Military Medical Research and Development program (CFDA 12.420) to develop an UNC13A antisense oligonucleotide treatment for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) from June 1, 2023 to May 31, 2025. The grant funding will support Acurastem's efforts to advance a novel gene therapy targeting UNC13A, a gene implicated in ALS and FTD, using antisense oligonucleotide...

The National Institute of Neurological Disorders and Stroke (NINDS) awarded a $391,903 Project Grant under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) to the Allen Institute to develop a new antisense oligonucleotide (ASO) therapeutic for Dravet syndrome, a severe epileptic encephalopathy. The grant will fund a two-phase project to optimize the ASO, establish its pharmacokinetics and pharmacodynamics, and validate a large-scale batch of the lead...